On June 10, 2026 Sumitomo Pharma America, Inc. (SMPA) reported that it has enrolled the required number of participants in its pivotal Phase 2 monotherapy trial for enzomenib, an investigational, oral selective menin inhibitor for the treatment of relapsed/refractory acute leukemia with KMT2A rearrangement, to allow for interim analysis. Achieving this milestone will enable SMPA to obtain results of the interim analysis by the end of calendar year 2026. These results will be promptly disclosed, with detailed data to be presented at an upcoming medical congress. If the primary endpoint is met, SMPA would then proceed with preparation for regulatory submission with the aim of obtaining approval in the US and Japan during FY2027.
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"Acute leukemias, especially at the relapsed/refractory stage, are profoundly difficult to treat, and are associated with very poor outcomes," said Tsutomu Nakagawa, Ph.D., President and Chief Executive Officer of SMPA. "Achieving this enrollment milestone is an important step in our efforts to bring a new, differentiated therapeutic option to acute leukemia patients and their families. My sincere thanks to the patients who are taking part in this study to further our scientific understanding of leukemia treatment."
In addition to this study, SMPA continues to enroll patients in its pivotal Phase 2 monotherapy study of enzomenib in patients with relapsed or refractory acute myeloid leukemia (AML) with NPM1 mutation.
About Leukemia
Leukemia is a type of cancer that forms in blood-forming tissue, characterized by the uncontrolled growth of blood cells, usually white blood cells, in the bone marrow. Acute leukemia, a form of leukemia, requires immediate treatment as blood cells multiply rapidly leading to a sudden onset of symptoms.1 Approximately 30% of patients with AML have NPM1 mutations,2 and 5%-10% of patients with AML have KMT2A rearrangements.3
About Enzomenib (DSP-5336)
Enzomenib is an investigational, oral, small molecule inhibitor of the menin and lysine (K)-specific methyltransferase 2A (KMT2A) protein interaction, a key interaction in acute leukemia and other tumor cell proliferation and growth. Menin is a scaffold nuclear protein that plays key roles in gene expression and protein interactions involved in many biological pathways, including cell growth, cell cycle, genomic stability, and hematopoiesis.4,5 In preclinical studies, enzomenib has shown selective growth inhibition in human acute leukemia cell lines with KMT2A rearrangements or NPM1 mutations.4,6 Enzomenib reduced the expression of the leukemia-associated genes HOXA9 and MEIS1 and increased the expression of the differentiation gene CD11b in human acute leukemia cell lines with KMT2A rearrangements and NPM1 mutation.7,8 The safety and efficacy of enzomenib is currently being clinically evaluated in a Phase 1/2 dose-escalation/dose-expansion study in patients with relapsed or refractory acute leukemia (NCT04988555) and the registrational Phase 2 Horizen-1 R/R mono AML/ALL (KMT2Ar + NPM1m) study. The FDA granted Orphan Drug Designation for enzomenib for the indication of acute myeloid leukemia in June 2022. The FDA granted Fast Track Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with KMT2Ar or NPM1m in June 2024. Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) granted Orphan Drug Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with KMT2Ar or NPM1m in September 2024.
(Press release, Sumitomo Dainippon Pharma, JUN 10, 2026, View Source [SID1234666545])